Mackenzie W Ferguson, Connor J Kennedy, Thulani H Palpagama, Henry J Waldvogel, Richard L M Faull, Andrea Kwakowsky
{"title":"Current and Possible Future Therapeutic Options for Huntington's Disease.","authors":"Mackenzie W Ferguson, Connor J Kennedy, Thulani H Palpagama, Henry J Waldvogel, Richard L M Faull, Andrea Kwakowsky","doi":"10.1177/11795735221092517","DOIUrl":"10.1177/11795735221092517","url":null,"abstract":"<p><p>Huntington's disease (HD) is an autosomal neurodegenerative disease that is characterized by an excessive number of CAG trinucleotide repeats within the huntingtin gene (<i>HTT).</i> HD patients can present with a variety of symptoms including chorea, behavioural and psychiatric abnormalities and cognitive decline. Each patient has a unique combination of symptoms, and although these can be managed using a range of medications and non-drug treatments there is currently no cure for the disease. Current therapies prescribed for HD can be categorized by the symptom they treat. These categories include chorea medication, antipsychotic medication, antidepressants, mood stabilizing medication as well as non-drug therapies. Fortunately, there are also many new HD therapeutics currently undergoing clinical trials that target the disease at its origin; lowering the levels of mutant huntingtin protein (mHTT). Currently, much attention is being directed to antisense oligonucleotide (ASO) therapies, which bind to pre-RNA or mRNA and can alter protein expression via RNA degradation, blocking translation or splice modulation. Other potential therapies in clinical development include RNA interference (RNAi) therapies, RNA targeting small molecule therapies, stem cell therapies, antibody therapies, non-RNA targeting small molecule therapies and neuroinflammation targeted therapies. Potential therapies in pre-clinical development include Zinc-Finger Protein (ZFP) therapies, transcription activator-like effector nuclease (TALEN) therapies and clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated system (Cas) therapies. This comprehensive review aims to discuss the efficacy of current HD treatments and explore the clinical trial progress of emerging potential HD therapeutics.</p>","PeriodicalId":15218,"journal":{"name":"Journal of Central Nervous System Disease","volume":null,"pages":null},"PeriodicalIF":2.6,"publicationDate":"2022-05-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9125092/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"42174445","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Viloxazine for Attention-Deficit Hyperactivity Disorder: A Systematic Review and Meta-analysis of Randomized Clinical Trials.","authors":"Alok Singh, Mahesh Kumar Balasundaram, Abhishek Singh","doi":"10.1177/11795735221092522","DOIUrl":"10.1177/11795735221092522","url":null,"abstract":"<p><strong>Background: </strong>Recently, the United States Food and Drug Administration (USFDA) approved viloxazine extended-release (ER) to manage attention-deficit hyperactivity disorder (ADHD) in pediatric patients of 6-17 years of age.</p><p><strong>Objective: </strong>To perform a meta-analysis to determine the safety and efficacy of viloxazine ER in the management of ADHD.</p><p><strong>Data source and methods: </strong>A literature search was performed through the databases Cochrane Library, PubMed, and clinicaltrials.gov, for a period from inception to August 2021, with the keywords: viloxazine, SPN-812, ADHD, and randomized clinical trials. The randomized controlled trials published in English language that analyzed the efficacy and safety were included. The risk of bias (RoB) was assessed by RoB tool. The outcomes included in this study were the proportion of patients with a 50% reduction in ADHD-Rating Scale-5 (ADHD-RS-5 responders) and improvement in CGI-I scale and the proportion of patients with at least one adverse event, the incidence of somnolence and Serious Adverse Events (SAEs).</p><p><strong>Results: </strong>This meta-analysis includes 1605 patients from five randomized clinical trials; all of the trials were at low risk of bias. Viloxazine group had more ADHD-RS-5 responders as compared to placebo; RR = 1.62; 95% CI = 1.36-1.93; <i>P</i> = <.00001. Significantly higher number of patients showed improved CGI-I score; RR = 1.53; 95% CI = 1.32-1.78; <i>P</i> = <.00001. A higher proportion of patients was observed with at least one adverse event (RR = 1.52; 95% CI = 1.24-1.85; <i>P</i> = <.0001), and somnolence (RR = 3.93; 95% CI = 2.11-7.31; <i>P</i> = <.0001) in viloxazine group. The incidence of SAEs was more in viloxazine group (RR = 2.98; 95% CI = .67-13.3; <i>P</i> = .15).</p><p><strong>Conclusions: </strong>Viloxazine was found to be significantly superior to placebo in both efficacy outcomes. Adverse events and somnolence were significantly more than the placebo. The incidence was SAEs was more in the viloxazine group but was not statistically significant.</p>","PeriodicalId":15218,"journal":{"name":"Journal of Central Nervous System Disease","volume":null,"pages":null},"PeriodicalIF":2.6,"publicationDate":"2022-05-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/35/ee/10.1177_11795735221092522.PMC9125110.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10250403","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
L. Diem, Livia Fregolente-Gomes, J. Warncke, H. Hammer, C. Friedli, N. Kamber, Simon Jung, S. Bigi, M. Funke-Chambour, A. Chan, C. Bassetti, A. Salmen, R. Hoepner
{"title":"Fatigue in Post-COVID-19 Syndrome: Clinical Phenomenology, Comorbidities and Association With Initial Course of COVID-19","authors":"L. Diem, Livia Fregolente-Gomes, J. Warncke, H. Hammer, C. Friedli, N. Kamber, Simon Jung, S. Bigi, M. Funke-Chambour, A. Chan, C. Bassetti, A. Salmen, R. Hoepner","doi":"10.1177/11795735221102727","DOIUrl":"https://doi.org/10.1177/11795735221102727","url":null,"abstract":"Introduction Post-COVID-19 syndrome affects approximately 10-25% of people suffering from COVID-19 infection, irrespective of initial COVID-19 severity. Fatigue is one of the major symptoms, occurring in 30-90% of people with post-COVID-19 syndrome. This study aims at describing factors associated with fatigue in people with Post-COVID-19 seen in our newly established Post-Covid clinic. Methods This retrospective single center study included 42 consecutive patients suffering from Post-COVID-19 syndrome treated at the Department of Neurology, University Hospital Bern, between 11/2020 and05/2021. Clinical phenomenology of Post-COVID-19 syndrome with a special focus on fatigue and risk factor identification was performed using Mann-Whitney U Test, Pearson Correlation, and Chi-Quadrat-Test. Results Fatigue (90.5%) was the most prevalent Post-COVID-19 symptom followed by depressive mood (52.4%) and sleep disturbance (47.6%). Fatigue was in mean severe (Fatigue severity scale (FSS) mean 5.5 points (95% Confidence interval (95CI) 5.1 - 5.9, range .9 - 6.9, n = 40), and it was unrelated to age, COVID-19 severity or sex. The only related factors with fatigue severity were daytime sleepiness and depressed mood. Conclusion Fatigue is the main symptom of the Post-COVID-19 syndrome in our cohort. Further studies describing this syndrome are needed to prepare the healthcare systems for the challenge of treating patients with Post-COVID-19 syndrome.","PeriodicalId":15218,"journal":{"name":"Journal of Central Nervous System Disease","volume":null,"pages":null},"PeriodicalIF":4.8,"publicationDate":"2022-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"47691164","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Victoria A Levasseur, Biao Xiang, Amber Salter, Dmitriy A Yablonskiy, Anne H Cross
{"title":"Stronger Microstructural Damage Revealed in Multiple Sclerosis Lesions With Central Vein Sign by Quantitative Gradient Echo MRI.","authors":"Victoria A Levasseur, Biao Xiang, Amber Salter, Dmitriy A Yablonskiy, Anne H Cross","doi":"10.1177/11795735221084842","DOIUrl":"10.1177/11795735221084842","url":null,"abstract":"<p><strong>Background: </strong>Multiple sclerosis (MS) lesions typically form around a central vein that can be visualized with FLAIR* MRI, creating the central vein sign (CVS) which may reflect lesion pathophysiology. Herein we used gradient echo plural contrast imaging (GEPCI) MRI to simultaneously visualize CVS and measure tissue damage in MS lesions. We examined CVS in relation to tissue integrity in white matter (WM) lesions and among MS subtypes.</p><p><strong>Objective: </strong>We aimed to determine if CVS positive lesions were specific to MS subtype, if CVS can be detected consistently among readers using the GEPCI method, and if there were differences in tissue damage in lesions with vs without CVS.</p><p><strong>Subjects and methods: </strong>Thirty relapsing-remitting MS (RRMS) subjects and 38 primary and secondary progressive MS (PMS) subjects were scanned with GEPCI protocol at 3T. GEPCI T2*-SWI images were generated to visualize CVS. Two investigators independently evaluated WM lesions for CVS and measured lesion volumes. To estimate tissue damage severity, total lesion volume, and mean lesion volume, R2t*-based tissue damage score (TDS) of individual lesions and tissue damage load (TDL) were measured for CVS+, CVS-, and confluent lesions. Spearman correlations were made between MRI and clinical data. One-way ANCOVA with age and sex as covariates was used to compare measurements of CVS+ vs CVS- lesions in each individual.</p><p><strong>Results: </strong>398 of 548 lesions meeting inclusion criteria showed CVS. Most patients had ≥40% CVS+ lesions. CVS+ lesions were present in similar proportion among MS subtypes. Interobserver agreement was high for CVS detection. CVS+ and confluent lesions had higher average and total volumes vs CVS- lesions. CVS+ and confluent lesions had more tissue damage than CVS- lesions based on TDL and mean TDS.</p><p><strong>Conclusion: </strong>CVS occurred in RRMS and PMS in similar proportions. CVS+ lesions had greater tissue damage and larger size than CVS- lesions.</p>","PeriodicalId":15218,"journal":{"name":"Journal of Central Nervous System Disease","volume":null,"pages":null},"PeriodicalIF":4.8,"publicationDate":"2022-03-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/9e/9f/10.1177_11795735221084842.PMC8973074.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10361599","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Francesco Pellegrini, Alessandra Cuna, Giovanni Prosdocimo
{"title":"Glaucomatous Type Cupping Caused by Internal Carotid Artery Compression: a Case Report.","authors":"Francesco Pellegrini, Alessandra Cuna, Giovanni Prosdocimo","doi":"10.1177/11795735221081588","DOIUrl":"https://doi.org/10.1177/11795735221081588","url":null,"abstract":"<p><p>A 71-year-old woman with a diagnosis of normal tension glaucoma (NTG) presented with complains of progressive visual loss in the right eye. Examination revealed features consistent with compressive optic neuropathy. Although brain magnetic resonance imaging (MRI) was initially interpreted as normal, re-evaluation disclosed a compression on the right optic nerve from the right internal carotid artery. We highlight the clinical differential diagnosis between NTG and compressive optic neuropathy. This case is a reminder that a compressive optic neuropathy may be caused by anatomical variation of normal intracranial structures.</p>","PeriodicalId":15218,"journal":{"name":"Journal of Central Nervous System Disease","volume":null,"pages":null},"PeriodicalIF":4.8,"publicationDate":"2022-02-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/d2/92/10.1177_11795735221081588.PMC8854233.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39938477","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Carmen Arenas-Cabrera, Pablo Baena-Palomino, Javier Sánchez-García, María Oliver-Romero, Yamin Chocrón-González, Manuel Caballero-Martínez
{"title":"Sleep-related hypermotor epilepsy with genetic diagnosis: description of a case series in a tertiary referral hospital.","authors":"Carmen Arenas-Cabrera, Pablo Baena-Palomino, Javier Sánchez-García, María Oliver-Romero, Yamin Chocrón-González, Manuel Caballero-Martínez","doi":"10.1177/11795735211060114","DOIUrl":"https://doi.org/10.1177/11795735211060114","url":null,"abstract":"<p><strong>Introduction: </strong>Sleep-related hypermotor epilepsy (SHE) is characterized by asymmetric tonic/dystonic posturing and/or complex hyperkinetic seizures occurring mostly during sleep. Experts agree that SHE should be considered a unique syndrome.</p><p><strong>Purpose: </strong>We present 8 cases of SHE for which a genetic diagnosis was carried out using a multigene epilepsy panel.</p><p><strong>Methods: </strong>We retrospectively screened familial and isolated cases of SHE in current follow-ups in our center.</p><p><strong>Results: </strong>We included 8 (5F/3M) patients, 5 of whom had a positive familial history of epilepsy. We identified a pathogenic mutation in <i>CHRNA4</i>, <i>CHRNB2</i>, and 3 different pathogenic changes in <i>DEPDC5</i>.</p><p><strong>Conclusions: </strong>Awareness of SHE needs to be raised, given its implications for finding an appropriate treatment, its relationship to cognitive and psychiatric comorbidities, and the opportunity to prevent the disorder in the descendants. We present our series with their clinical, radiological, electroencephalographic, and genetic characteristics, in which we found 3 pathogenic mutations in the <i>DEPDC5</i> gene but not previously reported in the literature. Identifying new pathogenic mutations or new genes responsible for SHE will facilitate a better understanding of the disease and a correct genetic counseling.</p>","PeriodicalId":15218,"journal":{"name":"Journal of Central Nervous System Disease","volume":null,"pages":null},"PeriodicalIF":4.8,"publicationDate":"2022-02-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/6e/5a/10.1177_11795735211060114.PMC8844731.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39934108","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Comparison of the effect and treatment sequence between a 2-week parallel repetitive transcranial magnetic stimulation and rehabilitation and a 2-week rehabilitation-only intervention during a 4-week hospitalization for upper limb paralysis after stroke: An open-label, crossover observational study.","authors":"Naoki Yamada, Kazumi Kashiwabara, Toru Takekawa, Midori Hama, Masachika Niimi, Takatoshi Hara, Satoshi Furumizo, Marika Tsuboi","doi":"10.1177/11795735211072731","DOIUrl":"https://doi.org/10.1177/11795735211072731","url":null,"abstract":"<p><strong>Background: </strong>NEURO<sup>®</sup> is a 2-week program that combines low-frequency repetitive transcranial magnetic stimulation (rTMS) and intensive occupational therapy (OT) to treat patients with chronic hemiparesis following stroke. The degree to which each element contributes to the improvement of upper limb function remains unclear. It has been suggested that low-frequency rTMS applied to a healthy cerebrum activates neural activity in the contralateral hemispheric area surrounding the lesion. Intensive OT performed in parallel to rTMS promotes the functional remodeling of the cerebrum to help with rehabilitation.</p><p><strong>Objectives: </strong>However, this has not been demonstrated using NEURO<sup>®</sup>. Therefore, we aimed to compare the effects of the NEURO<sup>®</sup> and OT-only protocols in patients with hemiparesis following stroke.</p><p><strong>Methods: </strong>Thirty-seven patients with upper limb paralysis following stroke were recruited and hospitalized for treatments and randomly divided into two groups. Group A consisted of 16 patients who underwent NEURO<sup>®</sup> for the first 2 weeks, and Group B consisted of 21 patients who underwent OT-only for the first 2 weeks. After 2 weeks of hospitalization, the treatments of Groups A and B were reversed for the subsequent 2 weeks of treatment. Improvement in upper limb motor function in Groups A and B at 2 and 4 weeks after the start of treatment was evaluated using the Fugl-Meyer Motor Assessment (FMA) and the Wolf Motor Function Test (WMFT).</p><p><strong>Results: </strong>Group A, who underwent NEURO<sup>®</sup> first during their initial 2-week hospitalization, showed significantly greater improvement than that in Group B, who underwent OT-only first (<i>P</i> = .041 for FMA and <i>P</i> < .01 for WMFT). At 4 weeks following the reversal of treatments, Group A who underwent NEURO<sup>®</sup> and then OT-only showed significantly greater improvement than that in Group B, who underwent OT-only followed by NEURO<sup>®</sup> (<i>P</i> = .011 for FMA and <i>P</i> = .001 for WMFT).</p><p><strong>Conclusion: </strong>Our findings indicate that rTMS facilitates neuromodulation when combined with OT, which leads to more effective rehabilitation than with OT alone (Trial registration: JMACCT (http://www.jmacct.med.or.jp/); trial ID JMA-IIA00215).</p>","PeriodicalId":15218,"journal":{"name":"Journal of Central Nervous System Disease","volume":null,"pages":null},"PeriodicalIF":4.8,"publicationDate":"2022-01-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/16/48/10.1177_11795735211072731.PMC8785323.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39963885","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Y. Aburto-Murrieta, Beatriz Méndez, J. Marquez-Romero
{"title":"Extended time window mechanical thrombectomy for pediatric acute ischemic stroke","authors":"Y. Aburto-Murrieta, Beatriz Méndez, J. Marquez-Romero","doi":"10.1177/11795735221098140","DOIUrl":"https://doi.org/10.1177/11795735221098140","url":null,"abstract":"Endovascular thrombectomy (EVT) for the treatment of acute ischemic stroke (AIS) remains an off-label procedure seldom utilized in the pediatric population; this holds especially true for patients presenting outside the standard 6-hour time window. In this review we describe the published literature regarding usage of the extended time window EVT in pediatric stroke. We searched PubMed for all pediatric AIS cases and case series that included patients treated with extended time window EVT. We found data from 38 cases found in 27 publications (15 case reports and 12 case series). The median age was 10 years; 60.5% males. The median NIHSS before EVT was 13 with a median time-to-treatment of 11 hours. The posterior circulation was involved in 50.0%. Stent retrievers were used in 68.5%, and aspiration in 13.2%. Angiographic outcome TICI ≥2B was achieved in 84.2%, whereas TICI˂2B was reported in 10.6%. A favorable clinical outcome (NIHSS score ≤4, modified Rankin score ≤1, or Pediatric Stroke Outcome measure score ≤1) occurred in 84.2%. Eight cases that did not report the clinical outcome employing a standardized scale described mild to absent neurological residual deficits. This study found data that supports that extended window EVT produces high recanalization rates and good clinical outcomes in pediatric patients with AIS. Nevertheless, the source materials are indirect and contain substantial inconsistencies with an increased risk of bias that amount to low evidence strength.","PeriodicalId":15218,"journal":{"name":"Journal of Central Nervous System Disease","volume":null,"pages":null},"PeriodicalIF":4.8,"publicationDate":"2022-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"46530526","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
G. Tsivgoulis, S. Deftereos, C. Gobbi, Elisabeth Gulowsen Celius, A. Kułakowska, G. Maniscalco, Irene Mendes, N. Grigoriadis
{"title":"Oral Cladribine in Patients who Change From First-Line Disease Modifying Treatments for Multiple Sclerosis: Protocol of a Prospective Effectiveness and Safety Study (CLAD CROSS)","authors":"G. Tsivgoulis, S. Deftereos, C. Gobbi, Elisabeth Gulowsen Celius, A. Kułakowska, G. Maniscalco, Irene Mendes, N. Grigoriadis","doi":"10.1177/11795735211069441","DOIUrl":"https://doi.org/10.1177/11795735211069441","url":null,"abstract":"Background Recently, the number of available disease modifying therapies for multiple sclerosis (MS) has increased. However, a proportion of patients treated with these agents continue to experience relapses and disease progression. Cladribine tablets, approved in 2017 for highly active relapsing MS, comprise a sparsely administered oral treatment which exerts its therapeutic effect through a reduction and subsequent repletion of the lymphocyte population. Purpose/Study Sample Here we describe the design of CLAD CROSS, a prospective, non-interventional, multicenter, Phase IV study in patients with a confirmed diagnosis of RRMS who switch from first-line disease modifying drugs (DMDs) to treatment with cladribine tablets in routine clinical practice. 242 adult patients will be recruited in 61 sites (6 countries) over 30 months and will be followed up for 2 years following prescription of cladribine tablets per the decision of the treating physicians. Research Design The primary endpoint is the change in annualized relapse rate (ARR) between the 12-month pre-baseline period and over the 12-month period before end of study. Secondary endpoints are the percentage of patients with 6-month disability progression or improvement at the end of the study, measured by the Expanded Disability Status Scale, Timed 25 Foot Walk and 9-Hole Peg Test scales and quality of life, treatment satisfaction, and healthcare resource utilization, measured through the MSIS-29, TSQM 1.4, and EQ-5D-3L scales, respectively. MRI lesions will be compared in the exploratory setting between the 12-month pre-baseline period, baseline, and at years 1 and 2. Adverse events will be monitored throughout the study. Interim analyses are pre-planned when 30% and 60% of patients will complete the 12-month follow-up visit. Conclusions CLAD CROSS will provide efficacy data on cladribine tablets, used as a follow-up treatment to first-line DMDs in the real-world setting, will further establish its safety profile and will collect information to support pharmacoeconomic studies.","PeriodicalId":15218,"journal":{"name":"Journal of Central Nervous System Disease","volume":null,"pages":null},"PeriodicalIF":4.8,"publicationDate":"2022-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"41545226","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
M. Auer, H. Hegen, A. Hotter, W. Löscher, K. Berek, Anne Zinganell, E. Fava, Paul Rhomberg, F. Deisenhammer, F. Di Pauli
{"title":"Recovery of Chronic Inflammatory Demyelinating Polyneuropathy on Treatment With Ocrelizumab in a Patient With Co-Existing Multiple Sclerosis","authors":"M. Auer, H. Hegen, A. Hotter, W. Löscher, K. Berek, Anne Zinganell, E. Fava, Paul Rhomberg, F. Deisenhammer, F. Di Pauli","doi":"10.1177/11795735221084837","DOIUrl":"https://doi.org/10.1177/11795735221084837","url":null,"abstract":"The chimeric anti-CD20 antibody rituximab has demonstrated good efficacy as an off-label treatment in chronic inflammatory demyelinating polyneuropathy (CIDP), while the humanized anti-CD20 antibody ocrelizumab has been approved for treatment of multiple sclerosis (MS), whereas there is no evidence for its use in CIDP so far. We present a patient suffering from CIDP and MS, both refractory to standard treatment and both showing marked improvement on ocrelizumab. To the best of our knowledge, this is a unique report of CIDP with an almost full electrophysiological recovery on ocrelizumab which could be considered as a potential treatment option for refractory CIDP.","PeriodicalId":15218,"journal":{"name":"Journal of Central Nervous System Disease","volume":null,"pages":null},"PeriodicalIF":4.8,"publicationDate":"2022-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"42620595","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}